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Feasibility and clinical integration of molecular profiling for target identification in pediatric solid tumors.
Pincez, Thomas; Clément, Nathalie; Lapouble, Eve; Pierron, Gaëlle; Kamal, Maud; Bieche, Ivan; Bernard, Virginie; Fréneaux, Paul; Michon, Jean; Orbach, Daniel; Aerts, Isabelle; Pacquement, Hélène; Bourdeaut, Franck; Jiménez, Irene; Thébaud, Estelle; Oudot, Caroline; Vérité, Cécile; Taque, Sophie; Owens, Cormac; Doz, François; Le Tourneau, Christophe; Delattre, Olivier; Schleiermacher, Gudrun.
Afiliación
  • Pincez T; Département d'oncologie pédiatrique, adolescents et jeunes adultes, Institut Curie, Paris, France.
  • Clément N; Service de Génétique, Institut Curie, Paris, France.
  • Lapouble E; Service de Génétique, Institut Curie, Paris, France.
  • Pierron G; Service de Génétique, Institut Curie, Paris, France.
  • Kamal M; Département d'oncologie médicale, Institut Curie, Paris et Saint Cloud, France.
  • Bieche I; Service de Génétique, Institut Curie, Paris, France.
  • Bernard V; Service de Génétique, Institut Curie, Paris, France.
  • Fréneaux P; Département de pathologie, Institut Curie, Paris, France.
  • Michon J; Département d'oncologie pédiatrique, adolescents et jeunes adultes, Institut Curie, Paris, France.
  • Orbach D; Département d'oncologie pédiatrique, adolescents et jeunes adultes, Institut Curie, Paris, France.
  • Aerts I; Département d'oncologie pédiatrique, adolescents et jeunes adultes, Institut Curie, Paris, France.
  • Pacquement H; Département d'oncologie pédiatrique, adolescents et jeunes adultes, Institut Curie, Paris, France.
  • Bourdeaut F; Département d'oncologie pédiatrique, adolescents et jeunes adultes, Institut Curie, Paris, France.
  • Jiménez I; Laboratoire de recherche translationnelle en oncologie pédiatrique, Institut Curie, Paris, France.
  • Thébaud E; Inserm U830 Laboratoire de Génétique et Biologie des Cancers, Institut Curie, Paris, France.
  • Oudot C; Département d'oncologie pédiatrique, adolescents et jeunes adultes, Institut Curie, Paris, France.
  • Vérité C; Laboratoire de recherche translationnelle en oncologie pédiatrique, Institut Curie, Paris, France.
  • Taque S; Service d'hématologie et oncologie pédiatrique, Centre Hospitalier Universitaire de Nantes, Nantes, France.
  • Owens C; Service d'onco-hématologie pédiatrique, Hôpital mère-enfant, Centre Hospitalier Universitaire de Limoges, Limoges, France.
  • Doz F; Unité d'hématologie pédiatrique, Groupe Hospitalier Pellegrin, Bordeaux, France.
  • Le Tourneau C; Département de médecine de l'enfant et de l'adolescent, Centre Hospitalier Universitaire de Rennes, Rennes, France.
  • Delattre O; National Pediatric Haematology/Oncology Centre, Our Lady's Children's Hospital, Dublin, Ireland.
  • Schleiermacher G; Département d'oncologie pédiatrique, adolescents et jeunes adultes, Institut Curie, Paris, France.
Pediatr Blood Cancer ; 64(6)2017 06.
Article en En | MEDLINE | ID: mdl-27896933
BACKGROUND: The role of tumor molecular profiling in directing targeted therapy utilization remains to be defined for pediatric tumors. We aimed to evaluate the feasibility of a sequencing and molecular biology tumor board (MBB) program, and its clinical impact on children with solid tumors. PROCEDURE: We report on a single-center MBB experience of 60 pediatric patients with a poor prognosis or relapsed/refractory solid tumors screened between October 2014 and November 2015. Tumor molecular profiling was performed with panel-based next-generation sequencing and array comparative genomic hybridization. RESULTS: Mean age was 12 ± 5.7 years (range 0.1-21.5); main tumor types were high-grade gliomas (n = 14), rare sarcomas (n = 9), and neuroblastomas (n = 8). The indication was a poor prognosis tumor at diagnosis for 16 patients and relapsed (n = 26) or refractory disease (n = 18) for the remaining 44 patients. Molecular profiling was feasible in 58 patients. Twenty-three patients (40%) had a potentially actionable finding. Patients with high-grade gliomas had the highest number of targetable alterations (57%). Six of the 23 patients subsequently received a matched targeted therapy for a period ranging from 16 days to 11 months. The main reasons for not receiving targeted therapy were poor general condition (n = 5), pursuit of conventional therapy (n = 6), or lack of pediatric trial (n = 4). CONCLUSIONS: Pediatric molecular profiling is feasible, with more than a third of patients being eligible to receive targeted therapy, yet only a small proportion were treated with these therapies. Analysis at diagnosis may be useful for children with very poor prognosis tumsors.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Sarcoma / Glioma / Neuroblastoma Tipo de estudio: Diagnostic_studies / Prognostic_studies Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Pediatr Blood Cancer Asunto de la revista: HEMATOLOGIA / NEOPLASIAS / PEDIATRIA Año: 2017 Tipo del documento: Article País de afiliación: Francia Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Sarcoma / Glioma / Neuroblastoma Tipo de estudio: Diagnostic_studies / Prognostic_studies Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Pediatr Blood Cancer Asunto de la revista: HEMATOLOGIA / NEOPLASIAS / PEDIATRIA Año: 2017 Tipo del documento: Article País de afiliación: Francia Pais de publicación: Estados Unidos